An Open-Label Extension Study of BPN14770 in Subjects With Fragile X Syndrome

NCT ID: NCT05367960

Last Updated: 2025-10-03

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE3
• Total Enrollment: 314 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-11-01
• Study Completion Date: 2027-12-31

Brief Summary

This is a 4-year, open-label extension (OLE) study for subjects completing one of two double-blind clinical trials with BPN14770, Study BPN14770-CNS-301 (in adult males) and Study BPN14770-CNS-204 (in adolescent males).

Detailed Description

This is a 4-year open-label extension (OLE) study for subjects completing one of two double-blind clinical trials with BPN14770, Study BPN14770-CNS-301 (in adult males) and Study BPN14770-CNS-204 (in adolescent males). The primary objective of this OLE is to assess the long-term safety and tolerability of BPN14770 in these subjects with fragile X syndrome (FXS) who were treated in one of those parent clinical trials.

Conditions

Fragile X Syndrome

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Study Drug (BPN14770)

25mg BID Study Drug BPN14770 (Adults) or 15mg BID Study Drug BPN14770 (Adolescents with body weight \<43 kg)

Group Type: OTHER

Zatolmilast/ BPN14770

Intervention Type: DRUG

25mg zatolmilast/BPN14770 (Adults) or 15 mg zatolmilast/BPN14770 (Adolescents \<43 kg)

Interventions

Zatolmilast/ BPN14770

25mg zatolmilast/BPN14770 (Adults) or 15 mg zatolmilast/BPN14770 (Adolescents \<43 kg)

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Subject has completed the Week 13 visit, whether on treatment or discontinued from treatment, from one of two parent clinical trials with

BPN14770:

1. Study 204

2. Study 301 Subjects who discontinued study treatment early due to AEs deemed related to study treatment by the investigator in one of the parent studies will NOT be eligible, regardless of whether the Week 13 visit was completed.

2. Subjects with a history of seizure disorder who are currently receiving treatment with antiepileptics must have remained seizure-free during the parent study. Any subject experiencing a seizure during the parent study is not eligible to continue into this long-term safety study.

3. Subject must be willing to practice barrier methods of contraception while on study, if sexually active. Abstinence is also considered a reasonable form of birth control in this study population.

4. Subject has a parent, legal authorized guardian, or consistent caregiver.

5. Subject and caregiver are able to attend the clinic regularly and reliably.

6. If subject is his own legal guardian, he is able to understand and sign an informed consent form to participate in the study.

7. For subjects who are not their own legal guardian, subject's parent/legally authorized guardian is able to understand and sign an informed consent form for their child to participate in the study.

8. If subject is not his own legal guardian, subject must provide assent for participation in the study if he has the cognitive ability to do so.

Exclusion Criteria

• 1. History of, or current cardiovascular, renal, hepatic, respiratory, gastrointestinal, psychiatric, neurologic, cerebrovascular, or other systemic disease that would place the subject at risk or potentially interfere with the interpretation of the safety, tolerability, or efficacy of the study treatment. Common conditions such as mild hypertension, etc. are allowed per the principal investigator's (PI) judgement as long as they are stable and controlled by medical therapy that is constant for at least 4 weeks before randomization.

2. Renal impairment, defined as serum creatinine >1.25×ULN per the latest available laboratory results from the parent study.

3. Cirrhosis, unstable chronic liver disease or acute liver disease. Hepatic impairment, defined as ALT or AST elevation > 2 × ULN per the latest available laboratory results from the parent study. Subjects with stable chronic hepatitis B on oral anti-viral therapy and subjects cured of chronic hepatitis C are allowed. Subjects with Gilbert syndrome are allowed.

4. Clinically significant abnormalities, in the investigator's judgement, in safety laboratory tests, vital signs, or ECG, as measured at the final visit of the parent study.

5. Substance abuse documented during the parent study.

6. Significant hearing or visual impairment that may affect the subject's ability to complete the test procedures.

7. Concurrent major psychiatric condition (eg, major depressive disorder, schizophrenia, or bipolar disorder) as confirmed by the investigator. Subjects with additional diagnosis of autism spectrum disorder or anxiety disorder will be allowed.

8. Subject has active diseases that would interfere with participation, such as acquired immunodeficiency disorder, hepatitis C, hepatitis B, or tuberculosis.

• Minimum Eligible Age: 9 Years
• Maximum Eligible Age: 45 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Tetra Discovery Partners

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Elizabeth Berry-Kravis, MD

Role: PRINCIPAL_INVESTIGATOR

Rush University Medical Center

Locations

Amnova Clinical Research

Irvine, California, United States

Thompson Autism & Neurodevelopment Center - CHOC

Orange, California, United States

MIND Institute UC Davis Medical Center

Sacramento, California, United States

Children's Hospital Colorado

Aurora, Colorado, United States

University of Miami

Miami, Florida, United States

Emory University School of Medicine

Atlanta, Georgia, United States

Rush University Medical Center

Chicago, Illinois, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

Kennedy Krieger Institute

Baltimore, Maryland, United States

Boston Children's Hospital

Boston, Massachusetts, United States

U Mass

Worcester, Massachusetts, United States

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Suburban Research Associates

Media, Pennsylvania, United States

Clinic for Special Children

Strasburg, Pennsylvania, United States

Greenwood Genetic Center

Greenville, South Carolina, United States

University of Utah and Primary Children's Hospital

Salt Lake City, Utah, United States

Countries

United States

Other Identifiers

BPN14770-CNS-302

Identifier Type: -

Identifier Source: org_study_id